317. Trifunctional protein deficiency
3 clinical trials,   8 drugs   (DrugBank: 4 drugs),   1 drug target gene,   1 drug target pathway

Searched query = "Trifunctional protein deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

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No.TrialIDDate_
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1NCT02517307
(ClinicalTrials.gov)
February 20162/7/2015Fatty Acid Oxidation Defects and Insulin SensitivityRole of Fatty Acid Oxidation Defects in Insulin SensitivityVery Long-chain Acyl-CoA Dehydrogenase Deficiency;Trifunctional Protein Deficiency;Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;Medium-chain Acyl-CoA Dehydrogenase Deficiency;Normal Volunteers;Carnitine Palmitoyltransferase II Deficiency, MyopathicDrug: Intralipid/Heparin;Drug: Glycerol/Saline;Drug: Hyperinsulinemic euglycemic clampOregon Health and Science UniversityNULLRecruiting18 YearsN/AAll50N/AUnited States
2NCT01461304
(ClinicalTrials.gov)
November 30, 201125/10/2011Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncAvailable1 MonthN/AAllUnited States
3NCT00840112
(ClinicalTrials.gov)
July 20109/2/2009Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated NeuropathyVitamin E Treatment for LCHAD Associated NeuropathyPeripheral Neuropathy;Mitochondrial Trifunctional Protein DeficiencyDietary Supplement: Vitamin E supplementOregon Health and Science UniversityNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Oregon State UniversityTerminated7 YearsN/ABoth1N/AUnited States